Combined Steroid and Cyclosporin as First-line Treatment in Adults With Primary Immune Thrombocytopenia

Peking University

Status and phase: Not yet enrolling; Phase 2

Conditions: Immune Thrombocytopenia

Treatments: Drug: Cyclosporine Oral Product; Drug: Prednisone

Study type: Interventional

Funder types: Other

Identifiers

NCT05459649

CSA-ITP

Details and patient eligibility

About

Randomized, open-label, multicenter study to compare the efficacy and safety of cyclosporin plus standard steroid compared to standard steroid monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Full description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 253 adults with ITP in China. Patients were randomized to cyclosporin plus standard steroid compared to standard steroid monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Enrollment: 253 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Newly-diagnosed, treatment-naive primary ITP;
2. Platelet counts <30×10^9/L;
3. Platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
4. Willing and able to sign written informed consent.

Exclusion criteria

1. Pregnant or lactating women;
2. Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
3. Received first-line and second-line ITP specific treatments (e.g., steroids, intravenous immunoglobulin, TPO-RAs, rhTPO, rituximab, etc);
4. Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
5. Severe medical condition (lung, heart, hepatic or renal disorder);
6. Patients who are deemed unsuitable for the study by the investigator.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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253 participants in 2 patient groups

Cyclosporin plus Steroid

Experimental group

Description:

Cyclosporin: started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count. Standard regimen of steroid for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.

Treatment:

Drug: Cyclosporine Oral Product

Drug: Prednisone

Standard steroid

Active Comparator group

Description:

Standard regimen for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.

Treatment:

Drug: Prednisone